With Data, Pfizer/Sangamo Step Forward in Hemophilia Gene Therapy Race
Summary
At the International Society on Thrombosis and Haemostasis meeting in Melbourne, Pfizer (NYSE: PFE) and partner Sangamo Therapeutics (NASDAQ: SGMO) disclosed the latest results from an early-stage study, Alta, of a gene therapy for hemophilia called SB-525. Pfizer and Sangamo reported one serious side effect: one patient had low blood pressure and a fever six hours after treatment, and was later discharged from the hospital. SB-525 is “still early but very intriguing, and merits close monitoring given [the BioMarin program’s] promising but admittedly imperfect product profile,” wrote Stifel analyst Paul Matteis in a research note. Hemophilia A is typically treated with two or three self-injections a week of replacement factor VIII, which helps patients prevent dangerous bleeds that can lead to joint damage and other health problems. SB-525, for instance, delivers less virus than valrox, which may be playing a role in its early results, wrote Jefferies analyst Raycroft.
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